A media release photo of Petri Polonen and Charles Mullighan.
Researchers from Children’s Hospital of Philadelphia (CHOP), St. Jude Children’s Research Hospital, and the Children’s Oncology Group have made a groundbreaking discovery about T-lineage acute lymphoblastic leukemia (T-ALL), a highly aggressive form of cancer.
Their study, published in Nature, has identified that genetic changes in non-coding regions of DNA, rather than protein-coding genes, are frequently responsible for driving T-ALL. This paradigm shift has significant implications for understanding and treating this devastating disease.
The research was supported by the Gabriella Miller Kids First Pediatric Research Program and the National Institutes of Health Common Fund.
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T-lineage acute lymphoblastic leukemia (T-ALL) is a rare but aggressive form of cancer that affects the T cells, a type of white blood cell. In T-ALL, immature T cells grow uncontrollably and crowd out healthy blood cells. The exact cause of T-ALL is unknown, but it is believed to be due to a combination of genetic and environmental factors.
Many children, adolescents, and young adult patients with T-ALL traditionally respond well to initial treatment. However, patients who relapse or have treatment-resistant disease often face a dire prognosis. Given the aggressive nature and rapid progression of the disease, and limited understanding of the genetic basis of T-ALL, researchers saw an urgent need for new and effective approaches to diagnosis and treatment.
“This paper is the first to transcend previous barriers and comprehensively profile the whole genome, uncovering critical insights in more than 1,300 children, adolescents and young adults with T-ALL,” said David T. Teachey, MD, an attending physician, Director of Clinical Research at the Center for Childhood Cancer Research at CHOP and Chair of the Acute Lymphoblastic Leukemia disease committee in the COG. “These findings are a significant clinical advancement, as the goal in treating T-ALL is to prevent relapse, which requires identifying the patients most at risk. This data now makes it possible to risk stratify patients with T-cell leukemia, identifying those with a high-risk of relapsing so we can treat them with newer or alternative medicines.”
The researchers explained prior studies were unable to identify important genetic changes in T-ALL, as they focused on the coding genome, the part of DNA that encodes proteins, the building blocks of cells. However, only 1% of DNA is coding, while the other 99% is termed non-coding.
Once considered useless, scientists now recognize that the non-coding region plays a key role in regulating biological processes. It signals the cell when to produce certain proteins, like a crossing guard aiding people to safely cross the street.
In this case, researchers studied more than 1,300 patients treated on the COG AALL0434 clinical trial and sequenced both the tumor and non-tumor genomes of each patient. While the researchers previously suspected that non-coding DNA in T-ALL played an important role, this study’s findings are the first ever to establish that at a large scale.
